The present invention relates to gene therapy tools that perform particularly well in the treatment of muscular dystrophy diseases, such as Duchenne muscular dystrophy (DMD).
This is based on the careful choice of antisense oligonucleotides (AON) making it possible to skip exon 53 of the dystrophin gene, conveyed by a recombinant adeno-associated viral vector (AAVr), and advantageously associated with a modified snRNA.